A government advisory committee on Tuesday recommended approval of a form of insulin that is inhaled rather than injected. The endorsement could lead to a new option for millions of Americans with diabetes and vindication for the persistent billionaire who spent a large amount of his fortune to develop the product.
Members of the committee, which advises the Food and Drug Administration, suggested that the inhaled insulin — called Afrezza and developed by the MannKind Corporation — would be useful for some patients, even if it might not be quite as effective as injected insulin.
“As an inhaled form of insulin, this represents a drug that will serve some patients that are not effectively served by currently available insulin,” Dr. Robert J. Smith, an endocrinologist at the Alpert Medical School at Brown University and the committee’s acting chairman, said after the vote.
The committee voted 13 to 1 that Afrezza was safe and effective enough for approval as a treatment for Type 1 diabetes, in which insulin is needed to survive. The endorsement for Type 2 diabetes, where insulin is usually one treatment option among many, was unanimous.
The votes were a bit of a surprise because the F.D.A.’s own staff had been critical in its review of Afrezza, calling it marginally effective and possibly risky. Also, MannKind had failed twice before in trying to win F.D.A. approval, requiring new clinical trials.
The committee, which met in Hyattsville, Md., was concerned that long-term exposure of the lungs to insulin could cause lung cancer. There were more cases of lung cancer among those who received Afrezza in clinical trials than in the control groups, but the numbers were small and far from definitive. MannKind is expected to undertake a long-term study to assess that risk.
The F.D.A. is supposed to decide whether to approve Afrezza by the middle of this month. The agency usually, but not always, follows the advice of its advisory committees.
MannKind, based in Valencia, Calif., is run by Alfred E. Mann, a highly successful aerospace and medical device entrepreneur still very active at age 88. MiniMed, a company he started that made insulin pumps for diabetics, was sold to Medtronic for about $3 billion in 2001. Mr. Mann also started companies that developed pacemakers and cochlear implants.
But there has been wonderment at Mr. Mann’s fascination and persistence with inhaled insulin. He has given his name to MannKind and spent much of his fortune — once estimated in the billions but recently put at $900 million by Forbes — sustaining it. He owns about 40 percent of MannKind, which has lost $2.3 billion since its inception.
Mr. Mann, in a statement, said he was pleased with the committee’s endorsement. “Diabetes is a major health problem in the United States,” he said, “and we are committed to bring Afrezza to the many patients who might benefit from this novel product.”
Trading in MannKind shares was halted on Tuesday.
Even if it wins approval, Afrezza might not be a commercial success. MannKind has yet to find the larger pharmaceutical company partner it has said it will need to market and sell the product.
The reason for the skepticism stems in part from Pfizer’s experience. To much fanfare, it won approval for an inhaled insulin called Exubera in 2006. But Exubera flopped, and less than two years later, Pfizer said it would stop selling the product and take a charge of $2.8 billion. Two insulin giants, Novo Nordisk and Eli Lilly, subsequently abandoned their own efforts to develop inhaled insulin.
But Mr. Mann soldiered on, saying the Afrezza inhaler was easier to use and less conspicuous than Pfizer’s inhaler, which was about the size of a tennis ball can. MannKind’s inhaler resembles a whistle and fits easily in the palm of a hand.
MannKind also claims its insulin is medically better because it takes effect faster than even rapid-acting injected insulins. That, it says, reduces the risk of hypoglycemia, a potentially life-threatening condition in which blood sugar drops too low.
However, the F.D.A. staff was skeptical, saying the lower incidence of hypoglycemia might be because Afrezza reduces blood sugar less than injected rapid-acting insulin.
In the main trial for Type 1 diabetes, the agency said, Afrezza was less effective than injected insulin in reducing blood sugar. Afrezza still met the statistical criterion for success in the trial, the agency said, but just barely.
In the clinical trial for those with Type 2 diabetes, Afrezza was better than an inhaled placebo, but the effect was “modest” compared with what was seen in the trials of many other diabetes drugs, the agency’s staff said.
MannKind argued, and many committee members seemed to agree, that inhaled insulin might appeal to patients with Type 2 diabetes who now delay starting on insulin, even though it would help control their condition. And some with Type 1 would welcome avoiding most of their daily injections.
“For many diabetics, these injections are exhausting,” said Rebecca W. Killion, who has diabetes and was the patient representative on the committee. “They’re frustrating, they’re disruptive and at this point inescapable.”